Welcome to Gene Therapy Net

Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials.

New Gene Therapy Shows Promise For Patients With Sickle Cell Disease

Posted on: 9 March 2019, source: CBS News
An experimental and innovative type of gene therapy is being used to attempt to cure sickle cell anemia. The director of the National Institutes of Health says the results of an NIH clinical trial on a gene therapy for sickle cell anemia "looks like a cure" for the painful and often deadly genetic disease that affects 100,000 Americans, with nearly all being African Americans. 60 Minutes follows the trial for more than a year, focusing on a Florida woman who now appears to be rid of sickle cell anemia. Dr. Jon LaPook's report will be broadcast on 60 Minutes, Sunday, March 10 at 7:00 p.m. ET/PT on CBS.

Gene Therapy Drug Approval to Skyrocket by 2025

Posted on: 16 January 2019, source: FDA
The FDA expects to approve 10 to 20 cell and gene therapy products a year within the next six years, and is hiring 50 more clinical reviewers to make it happen, the agency announced Jan. 15: "The FDA is witnessing a surge of cell and gene therapy products entering early development, evidenced by a large upswing in the number of investigational new drug (IND) applications. Based on this activity, we anticipate that the number of product approvals for cell and gene therapies will grow in the coming years, reflecting significant scientific advancement and the clinical promise of these new innovations."

Gene therapy for Crigler-Najjar syndrome - First patient treated in the European clinical trial

Posted on: 25 December 2018, source: Eurekalerts
Following patient recruitment and preliminary observation period, the first patient was injected by Prof. Labrune at Beclere Hospital in Clamart, France. Genethon, the AFM-Telethon laboratory, is sponsoring this multicenter international phase I/II trial, injecting intravenously an AAV vector that is able to transfer the UGT1A1 gene (coding for the production of bilirubin GT) into liver cells. A total of 17 patients will be treated over the next few months.

Chinese Researchers Claim to Have Genetically Engineered the First HIV-Immune Babies

Posted on: 28 November 2018, source: fortune.com
Researchers in China (Clinical project 'Safety and validity evaluation of HIV immune gene CCR5 gene editing in human embryos') used genetic engineering tools (CRISPR) to create twins theoretically immune to HIV, smallpox, and cholera, MIT Technology Review reported. The medical breakthrough is controversial, as many worry about eugenics and designer babies for the wealthy. The twins, named Lulu and Nana, according to lead scientist He Jiankui of Shenzhen in a YouTube video, were the result of in vitro fertilization (IVF). A few weeks old, they appear to be healthy. When they were a single cell, genetic surgery using a popular tool, CRISPR, “removed the doorway through which HIV enters to infect people.”

Watch the presentation of He Jiankui and discussion during Second International Summit on Human Genome Editing in Hongkong, Wednesday November 28th, 2018.

Spark Therapeutics' Luxturna Gets EU Approval for Vision Loss

Posted on: 26 November 2018, source: Nasdaq
Spark Therapeutics, Inc. announced that the European Commission has approved the marketing authorization application ("MAA") for its gene therapy, Luxturna (voretigene neparvovec). The MAA sought approval for the therapy in adult as well as pediatric patients as a one-time treatment for vision loss due to inherited retinal dystrophy caused by a genetic mutation in both copies of the RPE65-gene and who have enough viable retinal cells. The RPE65 mutations, a progressive disease, can lead to total blindness.

Gene therapy drugs step toward approval in Japan

Posted on: 14 November 2018, source: Nikkei Asia
Novartis has applied with Japanese authorities to have a gene therapy drug approved, a move that could give Japan at least two such drugs in the coming year, Nikkei has learned. The Switzerland-based pharmaceutical hopes to receive the all-clear in Japan for a neurological disorder treatment sometime next year. It has also applied for approval for the same drug in the U.S. and Europe. In the U.S., the drug is said to be prohibitively expensive -- as much as $4 million to $5 million per patient. The drug treats spinal muscular atrophy, and Novartis expects to be able to market the treatment as early as next year.

Infectivity of adeno-associated virus serotypes in mouse testis

Posted on: 7 November 2018, source: BMC Biotechnology
In a recent study, researchers from the Institute for Stem Cell Biology and Regenerative Medicine (InStem), Bengaluru, National Centre for Biological Sciences (NCBS), Bengaluru, Christian Medical College, Vellore and the Indian Institute of Technology, Kanpur, have described how a type of virus, called adeno-associated virus, behaves when injected into the mouse testis. The findings of the study, supported by the Department of Biotechnology (DBT) and InStem were published in the journal BMC Biotechnology.